Identification of genome safe harbor loci for human gene therapy based on evolutionary biology and comparative genomics
Marco A. Quezada‐Ramírez, Shannon Loncar, Matthew A. Campbell, Krishna Mohan Parsi, Robert James Gifford, Robert M. Kotin
The paper identifies genome safe harbor loci for gene therapy using evolutionary biology and comparative genomics, finding nine loci with sustained transgene expression in human hematopoietic stem cells.
iScience · Nov 1, 2025Peer-reviewedGenetics